Modalis Therapeutics

Genome Editing Tools

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Modalis Therapeutics is a Japan-based biotech company focused on developing gene therapies for rare genetic diseases that often lack effective treatments. They are utilizing a proprietary technology called CRISPR-GNDM® (guide nucleotide-directed modulation), which alters gene expression without permanent changes being made to the DNA sequence. This approach is unlike traditional CRISPR gene editing that cuts DNA. AAV vectors are used to deliver GNDM to the targeted cells. Advantages of this scalable technology include not requiring several doses, having a more sustained effect, and modifying the disease rather than symptom management alone. Indications within their pipeline include muscular, cardiovascular, and CNS disorders. Modalis also has facilities in Cambridge, MA.